• Yan Xu, Qian Wang, Hai-Tao Ren, Lin Qiao, Yao Zhang, Yun-Yun Fei, Yan Zhao, and Li-Ying Cui.
• Department of Neurology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China.
• J. Neurol. Sci. 2016 Nov 15; 370: 224-228.

AimsTo compare the efficacy and tolerability of azathioprine (AZA), mycophenolate mofetil (MMF), and cyclophosphamide (CTX) in patients with neuromyelitis optica spectrum disorder (NMOSD).MethodsWe performed a prospective cohort analysis of relapses, disability, and adverse events in NMOSD patients treated with AZA, MMF, or CTX (n=119, 38, and 41, respectively). All the patients were co-treated with oral prednisone.ResultsA significant reduction in relapse rate was found in patients taking AZA (p<0.001), MMF (p<0.001) or CTX (p=0.01). MMF was associated with a lower risk of relapse than AZA, but this difference was not statistically significant (p=0.08). AZA and MMF decreased the mean Expanded Disability Status Scale (EDSS) scores significantly (AZA: p=0.02; MMF: p=0.01), whereas CTX did not. Compared with AZA, MMF had a significantly lower risk of treatment discontinuation due to drug-related adverse events (p=0.02), whereas CTX had a comparable risk (p=0.35).ConclusionsMMF is a good first-line treatment option for NMOSD and AZA remains a valuable first-line drug if its side effects are tolerable while CTX can be a treatment option for patients who cannot tolerate AZA and MMF.Copyright © 2016 Elsevier B.V. All rights reserved.

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